Treatment Protocols

Treatment recommendations for myelodysplastic syndromes (MDS) are based on a patient’s Revised International Prognostic Scoring System score in addition to evaluation of a patient’s performance status. Currently, only a few FDA-approved therapies for MDS are available, including hypomethylating agents such as azacitidine and decitabine, iron chelators such as deferasirox, and lenalidomide.
[1] Special considerations and supportive care are also described below.
[2]

Allogeneic stem cell transplantation consultation should also be recommended

Allogeneic stem cell transplantation may be performed as initial therapy or following cytoreduction with any of the other therapies for MDS

Special considerations

See the list below:

Allogeneic stem cell transplantation is the only potentially curative therapy for MDS

Appropriate patients should be referred early for consultation with a transplant specialist, before extensive transfusion support, infectious complications, or transformation to AML

Generally, early transplantation is advocated for young patients who are IPSS INT-2–risk and high-risk patients
[15]

Consider referral for clinical trial participation at any stage of therapy

The regimens above have been tested in the frontline setting, and there is no standard of care at the inevitable time of frontline therapeutic failure

Clinical trial participation in this situation is highly recommended

When considering the choice of a particular therapeutic regimen, it is important to consider the time to best response; the duration of time to response is also critical in evaluating the success of therapies

Therapeutic regimens should generally not be changed in the absence of progression or toxicity unless an adequate trial of the current regimen has been undertaken

The median time to response for the therapies listed above is as follows:

Iron-chelating agents (deferoxamine, deferasirox) are available, but they have not been shown to decrease complications in MDS patients in prospective randomized trials. For this reason, many experts limit the use of these agents to patients likely to survive several years with transfusion dependence (eg, R-IPSS very-low-risk and low-risk patients).

Each iron-chelating agent has the following drawbacks: Deferoxamine is associated with inconvenient subcutaneous infusions, infusion-site reactions, and cataracts; deferasirox is an oral agent, but it is costly, requires monitoring of vision and renal and hepatic function, and is contraindicated in severe thrombocytopenia

References

Gotlib J. The hematologist: immunosuppression in myelodysplastic syndrome:where do we go from here? American Society of Hematology. [Full Text].

Silverman LR, McKenzie DR, Peterson BL, et al, and the Cancer and Leukemia Group B. Further analysis of trials with azacitidine in patients with myelodysplastic syndrome: studies 8421, 8921, and 9221 by the Cancer and Leukemia Group B. J Clin Oncol. 2006. 24:3895-903.